Cambridge Startup Hopes to Find Cure for US Boy’s Rare Disease

November 13, 2014


A Cambridge UK medical technology startup which matches safe drugs to rare diseases is working on a potential cure for a six-year-old American boy who is the first patient ever to be diagnosed with NGLY-1 deficiency and loss of function.

Healx is liaising with pharmaceutical entrepreneur and world-leading drug repositioning expert David Cavalla to try to help Bertrand Might in his fight against the rare congenital disorder.

NGLY1 deficiency is characterised by global developmental delay (often severe), neurological impairment, movement disorder and hypotonia (a state of low muscle tone) and can cause seizures, fibroses and heart damage.

Healx focuses on drug repositioning for rare diseases using sophisticated data analytics methods based on multiple chemical and biological data sources. It acts as a broker to match safe and tested drugs in particular against novel and non-obvious disease targets. In many cases Healx will match drugs to diseases where the prescribed therapies have a high failure rate.

The combined global markets are worth tens of billions of dollars but the patient outcomes could be priceless, which is the key driver for Healx.

CEO Tim Guilliams said specific details about the drugs concerned in Bertrand’s case were being kept confidential until more validation had been performed behind the scenes with the family and medical specialists.

“The Might family has gone through enough distress without having to endure needless false dawns,” Guilliams told Business Weekly.

“We have been absolutely amazed by their drive and determination. Matt and Cristina are a real inspiration to us. We are delighted to work with them and hope we can help drive a cure forward. Following further investigation of our identified marketed drugs, experimental validation will be the next key step.”

Launched in April as Healx3 by Cambridge and Oxford University alumni, the company swiftly engaged with two of the world’s largest biopharma companies.

The founders bring together their expertise in biopharma and computer science to create a strong team needed for this enterprise and continue to be advised by serial science & technology entrepreneurs in Cambridge, as well as Intellectual Property specialists.

The startup is backed by Accelerate Cambridge and Stevenage Bioscience Catalyst. They also cleverly managed to tap into the heart of Cambridge University’s bioscience expertise by collaborating with some of its world-leading academics, such as Andreas Bender from the Chemistry Department who has significant experience in chemical and biological data analysis for bioactive compound selection.

For now, Healx has chosen to grow organically. Guilliams said two investment offers of up to £100k had been postponed because the business preferred to remain independent from investors at this stage.

Healthy grant funding has enabled the company to start recruitment in earnest and the first full-time employee is starting this month; four part-time developers are already on board. Healx also acquired its first paying customers.

• PHOTOGRAPH SHOWS: Bertrand Might


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